DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Information source: Shire
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Gaucher Disease

Intervention: velaglucerase alfa (Biological)

Phase: Phase 3

Status: Completed

Sponsored by: Shire

Official(s) and/or principal investigator(s):
Bjorn Mellgard, M.D., Study Director, Affiliation: Shire

Summary

Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. The disease has been classified into 3 clinical subtypes based on the presence or absence of neurological symptoms and severity of neurological disease. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression. The primary objective of this study is to evaluate the safety of every other week dosing of velaglucerase alfa in Japanese patients with Gaucher disease. Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.

Clinical Details

Official title: A Multicenter, Open-Label Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

Number of Severe Adverse Events (SAE)

Number of Treatment Emergent Adverse Events (TEAE)

Development of Anti-velaglucerase Alfa Antibody

Number of Infusion- Related Adverse Events

Number of Patients With Concomitant Medication

Secondary outcome:

Change From Baseline in Hemoglobin Concentration

Change From Baseline in Platelet Count

Change From Baseline in Liver Volume, Normalized to Body Weight

Change From Baseline in Spleen Volume, Normalized to Body Weight

Change From Baseline in Plasma Chitotriosidase Levels

Change From Baseline in CCL18 Levels

Detailed description: Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Gaucher disease has been designated in the list of Specified Rare and Intractable Diseases by Specified Disease Treatment Research Program of Ministry of Health, Labor and Welfare (MHLW) as one of "lysosomal storage diseases" since 2001. Gaucher disease is also designated in the Medical Aid Program for Specified Categories of Chronic Pediatric Diseases. The prevalence of mutations and the phenotype of patients with Gaucher disease in Japan differs from that in non-Japanese populations. Some patients with type 1 Gaucher disease in Japan have more severe and progressive disease compared to non-Japanese patients and the disease is characterized by an earlier onset of symptoms. Velaglucerase alfa, a highly-purified form of the naturally occurring enzyme glucocerebrosidase, has been developed as an enzyme replacement therapy for Gaucher disease for the symptoms (anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone manifestation). The primary objective of this study is to evaluate the safety of every other week dosing of velaglucerase alfa in Japanese patients (naive or previously treated with imiglucerase) 2 years of age and older with Gaucher disease.

Eligibility

Minimum age: 2 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria:

- The patient has a documented diagnosis of Gaucher disease

- The patient is at least 2 years of age

- Female patients of child bearing potential must agree to use a medically acceptable

method of contraception at all times during the study

- The patient, the patient's parent(s) or legal guardian(s) has provided written

informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC)

- The patient must be sufficiently cooperative to participate in this clinical study as

judged by the Investigator Patients who are switched from imiglucerase ERT must meet the following additional criteria:

- Received treatment with imiglucerase for a minimum of 12 consecutive months

- Meet predefined limits for hemoglobin concentration and platelet counts

Patients naïve to treatment for Gaucher disease must meet the following additional criteria:

- Not received treatment for Gaucher disease (investigational or approved products)

within 12 months prior to study entry

- Have Gaucher disease related anemia and at least one of the following: moderate

splenomegaly or, Gaucher disease-related thrombocytopenia or Gaucher disease-related enlarged liver Exclusion Criteria:

- Treatment with any investigational drug or device within the 30 days prior to study

entry (time of informed consent); such use during the study is not permitted

- Positive for hepatitis B or hepatitis C.

- Non-Gaucher disease related anemia

- The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to

understand the nature, scope, and possible consequences of the study

- Significant comorbidity, as determined by the Investigator that might affect study

data or confound the study results

- The patient is unable to comply with the protocol or is unlikely to complete the

study, as determined by the Investigator

- The patient has experienced a severe (grade 3 or higher) infusion-related

hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)

- Currently receiving red blood cell growth factor, (eg, erythropoietin) or chronic

systemic corticosteroids in the last 6 months

- Patient has had a splenectomy or the patient has an active, clinically significant

spleen infarction within 12 months of screening

- Patient has worsening bone necrosis within 12 months of screening

- The patient is pregnant or lactating.

Locations and Contacts

Osaka City University Hospital, Osaka 545-0051, Japan

Hamamatsu University School of Medicine, Hamamatsu, Shizuoka 431-3192, Japan

The Jikei University School of Medicine, Minato-ku, Toyko 105-8461, Japan

Additional Information

Starting date: March 2012
Last updated: July 8, 2014

Page last updated: August 23, 2015

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017